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Characterizing SCN1A-Related Disorders Using Real-World Data Across 681 Patient-Years

2026-03-02 genetic and genomic medicine Title + abstract only
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SCN1A-related disorders are the single most common monogenic cause of epilepsy and represent a major focus of precision medicine efforts. In conjunction with existing prospective studies, the analysis of real-world data obtained during routine clinical care can expand upon the scale and duration of available data and contribute to the development of meaningful outcomes for clinical trials. Here, we leveraged real-world data to delineate the longitudinal disease history of 100 individuals with S...

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